Benzinga | Jun 1, 2021 08:25AM EDT

Legend Biotech Highlights New And Updated Data For Investigational BCMA CAR-T Ciltacabtagene Autoleucel For Treatment Of Relapsed Or Refractory Multiple Myeloma At 2021 ASCO And EHA Meetings

Longer-term results from the Phase 1b/2 CARTITUDE-1 study demonstrated 98 percent overall response rate, 80 percent stringent complete response rate and 66 percent progression free survival rate at 18 months with no new safety signals

First results from the CARTITUDE-2 study of cilta-cel in earlier treatment lines will be premiered

Legend Biotech Corporation (NASDAQ:LEGN) (Legend Biotech), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, announced today that new and updated results for ciltacabtagene autoleucel (cilta-cel), an investigational BCMA-directed CAR-T therapy for the treatment of relapsed or refractory multiple myeloma (RRMM), will be featured at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and at the European Hematology Association (EHA) Virtual Congress.

At a median follow-up of 18 months, updated results from the Phase 1b/2 CARTITUDE-1 study including 97 heavily pretreated patients with RRMM demonstrated an overall response rate (ORR) of 98 percent, with 80 percent of patients achieving a stringent complete response (sCR), highlighting a deepening response over time (from 67 percent reported at ASH 2020).1,2 The 18-month progression-free survival (PFS) rate was 66 percent (95 percent confidence interval [CI], 54.9-75.0) and overall survival rate (OS) rate was 81 percent (95 percent CI, 71.4-87.6). Patients had received a median of six prior lines of therapy (range, 3-18); 88 percent were triple-refractory and 42 percent were penta-refractory. Response rates were comparable (range, 95-100 percent) across prespecified subgroups, including number of prior lines of treatment, extramedullary plasmacytomas and cytogenetic risk.1

These data will be featured in an oral presentation at the 2021 ASCO Annual Meeting on Tuesday, June 8th (Abstract #8005) and as a poster presentation at the 2021 EHA Virtual Congress on Friday, June 11th (Abstract #EP964). The CARTITUDE-1 study supported the Biologics License Application for cilta-cel by Legend Biotech's collaborator, Janssen, which has been accepted for priority review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of November 29, 2021.

"What is remarkable about this study is that these patients, who had previously received multiple treatment regimens, have responded to cilta-cel without their disease progressing," said Saad Z. Usmani, M.D., Division Chief of Plasma Cell Disorders, Levine Cancer Institute. "These compelling results, paired with the manageable safety profile and sustained efficacy, demonstrate the potential of cilta-cel for patients with relapsed and refractory multiple myeloma."

Median time to first response was one month (range, 0.9--10.7 months) and responses deepened over time. Out of 61 minimal residual disease (MRD) evaluable patients, 92 percent achieved MRD negativity status at 10-5 at a median of one month (range, 0.8-7.7 months) post infusion.

Cilta-cel data showed a safety profile consistent with what has been previously reported and no new safety signals were observed with longer-term follow-up. The most common hematologic adverse events (AEs) observed in the CARTITUDE-1 study were neutropenia (96 percent); anemia (81 percent); thrombocytopenia (79 percent); leukopenia (62 percent); and lymphopenia (53 percent). Cytokine release syndrome (CRS) of any grade was observed in 95 percent of patients, with a median duration of four days (range, 1-97), and median time to onset of seven days (range, 1-12). Of the 92 patients with CRS, 95 percent experienced Grade 1/2 events and CRS resolved in 91 patients (99 percent) within 14 days of onset. There was no new incidence of neurotoxicity; neurotoxicity of any grade was observed in 21 percent (n=20) of patients, with Grade 3 or higher neurotoxicity observed in 10 percent (n=10) of patients.

"We are excited to share these latest results from the CARTITUDE-1 study which continue to show deep and sustained responses in patients who have been treated with cilta-cel," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "At Legend Biotech, we are continuing our efforts to build a robust pipeline of next-generation cell therapies with the potential to address unmet needs. We look forward to our continued collaborative efforts with Janssen as we to bring this personalized treatment to patients, pending regulatory approvals."